Center for Oncology Hematology Outcomes Research and Training (COHORT), Division of Hematology Oncology, University of California, Davis School of Medicine, Sacramento, CA.
University of California, Davis Comprehensive Cancer Center, Sacramento, CA.
J Natl Cancer Inst. 2019 Jan 1;111(1):78-85. doi: 10.1093/jnci/djy073.
Autologous hematopoietic stem cell transplant (aHSCT) is an efficacious treatment for newly diagnosed multiple myeloma patients. However, as rapid advances have resulted in other highly efficacious and less intensive therapies, the role of aHSCT has been questioned.
We utilized population-based data to identify 13 494 newly diagnosed patients younger than age 80 years between 1998 and 2012. Patient characteristics of aHSCT and non-aHSCT groups were balanced using inverse probability weighting of a propensity score predicting aHSCT use. Multivariable models adjusted for baseline comorbidities, demographics, and socioeconomic status estimated the adjusted hazard ratio (aHR) and 95% confidence intervals (CIs) of death.
Twenty point eight percent (2807) of patients underwent aHSCT, and this rate increased over time from 15.4% in 1998-2002 to 23.9% in 2008-2012. aHSCT was utilized among 37.6% and 11.5% of patients younger than age 60 years and 60 to 79 years, respectively. The median time to aHSCT was 9.4 months, and 89% of all aHSCTs occurred within two years of diagnosis. The median overall survival from time of aHSCT was 72.9 months (95% confidence interval [CI] = 68 to 78). Autologous HSCT at any time was associated with improved survival (aHR = 0.83, 95% CI = 0.75 to 0.92). Among aHSCT recipients, transplant more than 12 months after diagnosis (vs ≤12 months) was associated with worse survival (aHR = 1.33, 95% CI = 1.16 to 1.51). The positive effect of aHSCT on overall survival was similar across study time periods and age groups.
In the era of highly efficacious induction therapies, aHSCT remained infrequently used but continued to be associated with improved survival for multiple myeloma patients and should be considered for newly diagnosed patients.
自体造血干细胞移植（aHSCT）是新诊断多发性骨髓瘤患者的有效治疗方法。然而，随着快速进展带来了其他高效且强度较低的治疗方法，aHSCT 的作用受到了质疑。
我们利用基于人群的数据，确定了 1998 年至 2012 年间年龄小于 80 岁的 13494 名新诊断的患者。通过预测 aHSCT 使用的倾向评分的逆概率加权来平衡 aHSCT 组和非 aHSCT 组的患者特征。多变量模型调整了基线合并症、人口统计学和社会经济状况，估计了死亡的调整后危险比（aHR）和 95%置信区间（CI）。
28.07%（2807 人）的患者接受了 aHSCT，并且该比例随着时间的推移从 1998-2002 年的 15.4%增加到 2008-2012 年的 23.9%。年龄小于 60 岁和 60-79 岁的患者中，分别有 37.6%和 11.5%接受了 aHSCT。aHSCT 的中位时间为 9.4 个月，所有 aHSCT 中有 89%在诊断后两年内进行。从 aHSCT 开始的中位总生存时间为 72.9 个月（95%CI=68-78）。任何时间的自体 HSCT 均与生存改善相关（aHR=0.83，95%CI=0.75-0.92）。在接受 aHSCT 的患者中，与诊断后 12 个月内（≤12 个月）相比，12 个月后（>12 个月）进行移植与生存较差相关（aHR=1.33，95%CI=1.16-1.51）。aHSCT 对总生存的积极影响在不同研究时间段和年龄组中相似。
在高效诱导治疗的时代，aHSCT 的使用仍然很少见，但继续与多发性骨髓瘤患者的生存改善相关，应考虑用于新诊断的患者。
